Research & Development
Alnylam Submits CTA Application for ALN-HSD, an Investigational RNAi Therapeutic for the Treatment of Nonalcoholic Steatohepatitis
5 August 2020 - - US-based RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has submitted a clinical trial authorization application to The Medicines and Healthcare Products Regulatory Agency in the United Kingdom to initiate a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of nonalcoholic steatohepatitis, the company said.

The company plans to initiate a Phase 1 study in late 2020, upon obtaining MHRA approval.

ALN-HSD is an investigational, subcutaneously administered RNAi therapeutic targeting HSD17B13 in development in collaboration with Regeneron Pharmaceuticals for the treatment of nonalcoholic steatohepatitis.

ALN-HSD utilizes Alnylam's Enhanced Stabilization Chemistry Plus GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index.

The safety and efficacy of ALN-HSD have not been evaluated by the FDA, EMA or any other health authority.

Nonalcoholic steatohepatitis is a highly prevalent chronic liver disease in which inflammation and liver cell injury are caused by accumulation of hepatic fat. NASH is a subset of a group of conditions called nonalcoholic fatty liver disease (NAFLD) that can lead to progressive fibrosis, cirrhosis, and hepatocellular carcinoma.

Comorbidities include obesity, metabolic syndrome, and type 2 diabetes. Approximately 16 m people in the US live with NASH, with prevalence of the disease increasing due to rising rates of obesity.

NASH is projected to be the leading indication for liver transplants in developed countries within the next 10 years. There are currently no approved medical therapies for NASH.

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development.